July 14, 2020

The U.S. Food and Drug Administration has released updated information to help researchers and animal drug sponsors navigate the pathway to approval for animal drugs intended for minor uses and minor species (MUMS).

The FDA’s mission is to protect and promote the health of both human and animals, including those animals that are considered “minor species” – animals other than those typically thought of as livestock (cattle, pigs, chickens, turkeys) or companions (dogs, cats, horses). Minor species are animals that may not be quite so common – such as sheep, goats, and ferrets, but also zoo animals, wildlife, and insects like honey bees.

Similarly, the FDA has a responsibility to facilitate approval of treatments for conditions that are relatively rare in the major species, or which occur in small numbers or in limited geographic areas. Some examples of minor uses in major species include various cancers in dogs or regional occurrences of diseases, such as outbreaks of blackhead in turkey flocks in the Southern U.S.

Because MUMS drugs, by definition, are for use in relatively small markets, there is less economic incentive for a drug company to invest in the research and development necessary to bring a product to market via the traditional new animal drug approval process. Congress took action to address the disparity of products available to treat these animals by passing the Minor Use and Minor Species Animal Health Act of 2004 (the MUMS act). This act allows for alternative legal pathways to the marketplace for MUMS drugs (indexing and conditional approval) and also provides incentives, such as exclusive marketing rights and grant eligibility (via designation), for researchers and companies to overcome the financial roadblocks they face in producing animal drugs for limited markets.

In certain cases, a minor species drug is intended for use in species that are too rare or too varied to be the subject of adequate and well-controlled studies in support of a drug approval. The Index of Legally-Marketed Unapproved New Animal Drugs for Minor Species (the Index) provides an alternative FDA review process to affirm the safety and effectiveness of unapproved new animal drugs for use in non food-producing minor species. Minor use drugs are not eligible for the Index. There are currently 14 entries listed on the Index, for use in animals such as bison, elephants, raptors, ferrets and ornamental finfish. In 2018, the FDA issued GFI #210, entitled “The Index of Legally Marketed Unapproved New Animal Drugs for Minor Species” to provide detailed information about the program. 

Sponsors of MUMS drugs may pursue conditional approval of their products, which allows the marketing of a MUMS drug when all requirements for its approval have been met except the “substantial evidence” standard for effectiveness. Instead, conditional approval requires these drugs to meet a “reasonable expectation of effectiveness” standard. Conditional approval allows MUMS drugs to reach the market sooner, while the drug’s sponsor continues to collect the effectiveness data needed to support a full approval. 

The MUMS Designation program provides incentives similar to those available for human Orphan Products. Sponsors who are granted MUMS designation status are eligible to apply for grants to defray the costs of safety and effectiveness studies in support of the drug approval. They are also given 7 years of exclusive marketing rights beginning on the day of approval or conditional approval. 

Many of those seeking approval for MUMS drugs are new to the new animal drug approval process. The draft guidance released today differs from previous versions in that it provides a chronological “playbook” of the new animal drug approval process for MUMS drugs and explains the various scenarios that might arise along the way. This guidance also explains how to navigate the various components of the MUMS program so that sponsors can use them to their benefit when pursuing drug approval.  For sponsors of products intended for use in aquaculture, the guidance describes special considerations that apply.

FDA has also provided the following presentations to provide detailed information on the revised draft guidance.

• Part 1: Introduction and Overview
• Part 2: Minor Use Determinations and MUMS Designation Incentives
• Part 3: New Animal Drug Approval Process
• Part 4: Special Considerations for Aquatic Species

Draft Guidance for Industry #61, entitled “Special Considerations, Incentives, and Programs to Support the Approval of New Animal Drugs for Minor Uses and for Minor Species,” will be available for comment for 120 days, starting July 15, 2020. Comments will be accepted at any time but should be submitted no later than November 12, 2020, to ensure that the FDA takes the information into consideration before making further decisions on this issue.

To electronically submit comments to the docket, visit www.regulations.gov and type FDA-1997-D-0444 in the search box. For assistance in submitting electronic comments, please see regulations.gov help.

To submit comments to the docket by mail, use the following address. Be sure to include docket number FDA-1997-D-0444 on each page of your written comments.

Dockets Management Staff
HFA-305
Food and Drug Administration
5630 Fishers Lane, Room 1061
Rockville, MD 20852

For more information:

• Draft Guidance for Industry #61 Special Considerations, Incentives, and Programs to Support the Approval of New Animal Drugs for Minor Uses and for Minor Species
• FDA In Brief: FDA Revises Draft Guidance on Animal Drugs Intended for Minor Uses and Minor Species
• Guidance: Special Considerations, Incentives, and Programs to Support the Approval of New Animal Drugs for Minor Uses and for Minor Species
• Regulations.gov: please note that the public comment period opens on July 15, 2020